Beam Therapeutics' BEAM-301 eliminates starch supplementation in two patients in early trial
BEAM•Beam Therapeutics reported initial Phase 1/2 results for BEAM-301 in six patients with glycogen storage disease type Ia. Two of three patients receiving 40 mg achieved 15 hours of controlled fasting, eliminating daily starch supplementation at their latest assessments; four of six patients experienced serious treatment-related adverse events.
1. Initial trial results
Beam Therapeutics reported initial Phase 1/2 results for BEAM-301 in six patients with glycogen storage disease type Ia on Oct. 27, 2026. Two of three patients receiving 40 mg achieved 15 hours of controlled fasting at their latest assessments, eliminating daily starch supplementation. At Day 36, median fasting tolerance rose from 2.5 to 15 hours with 40 mg, compared with 2.9 to 4.3 hours with 20 mg. Four of six patients experienced serious treatment-related adverse events; no dose-limiting toxicities occurred. Enrollment in the 60 mg cohort is underway, and the company plans FDA discussions on a potential approval pathway.
